A child's growth is dependent on the proper functioning of the growth plate, a specialized cartilage structure located at the end of long bones and within the vertebrae. The primary function of the growth plate is to generate new cartilage, which is then converted into bone tissue and results in the lengthening of bones (i.e., the growth of the child). Mutations in one or more of the many genes that control growth plate function can cause severe skeletal growth disorders in children.
Current treatments for skeletal cartilage disorders are limited. One method of treatment involves the administration of recombinant growth hormone, but the results are less than optimal and systemic treatment using growth hormone carries a risk of increased intracranial pressure, slipped capital femoral epiphysis, insulin resistance, and possibly type II diabetes mellitus. Thus, there is a need for therapeutic agents and treatment methods that avoid the systemic risks of current therapies.